Paroxysmal Nocturnal Hemoglobinuria (PNH)
Treatment and Management of Aplastic Anemia and PNH
Advances in Paroxysmal Nocturnal Hemoglobinuria, AAMDSIF 2026 International Bone Marrow Failure Scientific Symposium
Recording from the AAMDSIF 2026 International Bone Marrow Failure Scientific Symposium, Co-Chairs Austin Kulasekarararaj, MBBS, and Pedro Prata, MD, PhD PNH Updates for 2026 - Regis Peffault de LaTour, MD, PhD Recognition and management of breakthrough hemolysis - Bruno Fattizzo…
Good job, Good health, Good life. Then: Aplastic Anemia, Followed by PNH. Daniel’s Story
In 2009, Daniel had a good job in accounting in Sydney, Australia. He had a structured, organized, healthy lifestyle. He had a devoted partner (whom he later married) and was studying for the Chartered Accountancy qualification. He rode his bike to work and played soccer. He was planning a good life ahead.
One day at the office, the receptionist pinched his arm. The next day, he saw a huge welt in the same spot. Because a colleague urged him to get it checked out, he did.
Health-Related Quality of Life, AAMDSIF 2026 International Bone Marrow Failure Scientific Symposium
Non-Transplant Therapies for Marrow Failure, AAMDSIF 2026 International Bone Marrow Failure Scientific Symposium
Clonality in Acquired Bone Marrow Failure, PNH, and VEXAS, AAMDSIF 2026 International Bone Marrow Failure Scientific Symposium
Urge Your U.S. Senators to Support the Bipartisan Letter to Increase Medical Research Accounts in the DoD
Imputing causality and clonal dynamics from single-cell transcriptomics in paroxysmal nocturnal hemoglobinuria
Abstract Paroxysmal nocturnal hemoglobinuria (PNH) originates from hematopoietic stem cells (HSCs) harboring somatic mutations in the phosphatidylinositol glycan class A (PIGA) gene. Clonal expansion of PIGA-mutated cells occurs uniquely in the setting of bone marrow (BM) failure…
Real-World Effectiveness and Safety of Ravulizumab in Patients With Paroxysmal Nocturnal Hemoglobinuria: Evidence From the International PNH Registry
Ravulizumab, a second-generation complement component 5 inhibitor (C5i) derived from eculizumab eculizumab: